• Improvements of 50% or more in total symptom score (TSS50) observed quickly and sustained
• Meaningful spleen volume reductions (SVR) observed and maintained
• Safety and tolerability of amsulostat shown out to 52 weeks

Syntara Limited (ASX:SNT), a clinical-stage drug development company, announced top-line data from the Phase 2a clinical trial evaluating amsulostat (200 mg BID) in combination with ruxolitinib (RUX) for the treatment of myelofibrosis (MF). The open-label study evaluated the safety and efficacy of amsulostat over 52 weeks in patients with intermediate-2 or high-risk MF who had been treated with RUX for an average of three years. Highlights include: Improvements of 50% or more in total symptom score (TSS50) were observed quickly (as early as 12 weeks) and were sustained, with 73% of patients achieving TSS50 at Week 24 or beyond. Meaningful spleen volume reductions (SVR) were observed at 24 weeks and maintained thereafter, with 44% of evaluable patients achieving SVR25 at Week 24 or beyond. The safety and tolerability of amsulostat, now shown out to 52 weeks, together with the increasing size and durability of clinical benefit seen beyond 24 weeks, compares very favorably with other drugs in development. Of the 7 patients who completed 52 weeks of treatment, 6 chose to continue on amsulostat through named patient supply, and 3 had a minor anaemia response. The positive results allow for the next stage of amsulostat clinical development and partnership engagement, supported by appointments of global strategic, clinical and commercial advisors.

The study results confirm the potential of amsulostat as a novel therapy for myelofibrosis, with sustained improvements in symptom burden and spleen volume, and a favorable safety and tolerability profile.

Syntara plans to engage with regulators and potential partners on the pathway forward for amsulostat's clinical development in myelofibrosis.